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MicroRNA-1 (miR-1) is a highly conserved, muscle-specific non-coding RNA that plays a critical role in the development and physiological maintenance of cardiac and skeletal muscle. It is encoded by two distinct genes, MIR1-1 and MIR1-2, which produce an identical mature sequence that regulates gene expression post-transcriptionally by binding to the 3' untranslated regions (UTRs) of target mRNAs (Source: 1.3.1, 1.4.1). In the heart, miR-1 is essential for cardiogenesis, sarcomere formation, and the regulation of cardiac conduction, while its dysregulation is linked to conditions such as myocardial infarction, arrhythmias, and heart failure (Source: 1.1.2, 1.2.3). Beyond its role in muscle, miR-1 acts as a potent tumor suppressor in various cancers, including lung, prostate, and colorectal cancer, where it inhibits cell proliferation, migration, and invasion by targeting oncogenes like MET and HDAC4 (Source: 1.3.1, 1.4.1, 2.3.3). Although no miR-1-targeted therapies are currently FDA-approved, experimental strategies such as miR-1 mimics for cancer treatment and antagomirs for cardiovascular protection are under investigation (Source: 1.1.1, 1.1.4). Circulating miR-1 has also emerged as a promising non-invasive biomarker for the early diagnosis of acute myocardial infarction and monitoring disease progression in muscular dystrophies (Source: 1.3.3, 2.3.2).
Post-transcriptional gene silencing through sequence-specific binding to the 3' untranslated regions (UTRs) of target mRNAs, leading to mRNA degradation or translational repression of genes such as Bcl-2, HDAC4, MET, and CXCR4 (Source: 1.1.1, 1.3.1, 2.1.3).
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